Clinical trial
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
NCT04221451 is registered in ClinicalTrials.gov with Genzyme, a Sanofi Company as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
- Lead sponsor
- Genzyme, a Sanofi Company
- Phase
- Phase 3
- Overall status
- Terminated
- Start date
- June 29, 2020
- Completion date
- December 26, 2024
- Enrollment
- 75 (actual)
- Sites
- 23
Conditions
As the registry lists them; a condition with its own page is linked.